Design & Rationale of the ATA-001-FKRP Gene Therapy Study in Fukutin- Related Protein Limb-Girdle Muscular Dystrophy (LGMDR9) Patients

Time: 12:00 pm


  • Atamyo’s trial uses an optimized gene therapy construct, ATA-100, a recombinant viral vector composed of the viral capsid of the adeno-associated virus serotype 9 (AAV9) and a codon-optimized complementary DNA (cDNA) sequence encoding the human FKRP under the transcriptional control of a human muscle-specific promoter
  • Safety, tolerability, effect on muscular, cardiac, and quality of life are secondary endpoints. About ten clinical sites in the US and Europe will participate in this trial
  • Safety status of the first patients treated in the dose escalation phase of the study will be presented